PMID- 31814747
OWN - NLM
STAT- PubMed-not-MEDLINE
LR  - 20231019
IS  - 1178-6981 (Print)
IS  - 1178-6981 (Electronic)
IS  - 1178-6981 (Linking)
VI  - 11
DP  - 2019
TI  - Budget Impact Of Eltrombopag As First-Line Treatment For Severe Aplastic Anemia 
      In The United States.
PG  - 673-681
LID - 10.2147/CEOR.S226323 [doi]
AB  - BACKGROUND: Severe aplastic anemia (SAA) is a rare autoimmune condition resulting 
      in low blood cell counts across lineages. Immunosuppressive therapy (IST) has 
      demonstrated low response, toxicity, and risk of transformation. In a Phase I/II 
      trial, the addition of eltrombopag to first-line IST increased response rates 
      relative to an IST-only historical cohort. METHODS: A model was developed to 
      estimate the budget impact of treating SAA with eltrombopag-based therapy from a 
      US private healthcare system perspective. A simulated cohort of newly diagnosed 
      SAA patients based on the total US population received 6 months of IST +/- 
      eltrombopag and were followed for 1 year, with mutually exclusive patient cohorts 
      entering in years 1, 2, and 3. The model assessed the budget impact of first-year 
      treatment for each cohort without considering subsequent years. At 6 months, 
      responders in either arm received maintenance therapy (low-dose cyclosporine), 
      and non-responders received 6 months of second-line eltrombopag monotherapy. 
      Costs considered included first-line, maintenance, and second-line therapy, 
      administration, routine care, mortality, and adverse events (AEs). All cost data 
      were reported in 2018 US dollars. RESULTS: The annual incidence of aplastic 
      anemia was 0.000234%, with 83.8% of cases assumed to be SAA. Based on trial data, 
      94% of patients receiving eltrombopag and IST responded versus 66% of patients 
      receiving IST, with a 0.3% reduction in the annual risk of mortality for the 
      eltrombopag + IST group. Use of first-line eltrombopag in a model SAA population 
      based on the total US population increased overall costs by $50 million over 3 
      years. First-line drug costs accounted for an increase of $69 million, while 
      improved response produced $19 million in secondary therapy cost savings. 
      Sensitivity analyses confirmed the robustness of the analysis. CONCLUSION: High 
      response rates combined with reduced rescue medication use and mortality in 
      patients treated with eltrombopag and IST mediated higher medication costs.
CI  - (c) 2019 Tremblay et al.
FAU - Tremblay, Gabriel
AU  - Tremblay G
AD  - Purple Squirrel Economics, New York, NY, USA.
FAU - Said, Qayyim
AU  - Said Q
AD  - Novartis Pharmaceutical Corporation, East Hanover, NJ, USA.
FAU - Roy, Anuja Nidumolu
AU  - Roy AN
AD  - Novartis Pharmaceutical Corporation, East Hanover, NJ, USA.
FAU - Cai, Beilei
AU  - Cai B
AD  - Novartis Pharmaceutical Corporation, East Hanover, NJ, USA.
FAU - Ashton Garib, Shan
AU  - Ashton Garib S
AD  - Purple Squirrel Economics, New York, NY, USA.
FAU - Hearnden, Jaclyn
AU  - Hearnden J
AD  - Purple Squirrel Economics, New York, NY, USA.
FAU - Forsythe, Anna
AU  - Forsythe A
AD  - Purple Squirrel Economics, New York, NY, USA.
LA  - eng
PT  - Journal Article
DEP - 20191112
PL  - New Zealand
TA  - Clinicoecon Outcomes Res
JT  - ClinicoEconomics and outcomes research : CEOR
JID - 101560564
PMC - PMC6858800
OTO - NOTNLM
OT  - United States
OT  - budget impact analysis
OT  - costs
OT  - eltrombopag
OT  - severe aplastic anemia
OT  - thrombopoietin receptor agonist
COIS- Qayyim Said and Beilei Cai are employees of Novartis Pharmaceutical Corporation. 
      Anuja Nidumolu Roy received services related to preparation and submission of 
      manuscript from Purple Squirrel Economics, during the conduct of the study and is 
      an employee of Novartis and owns its stock. Gabriel Tremblay, Shan Ashton Garib, 
      Jaclyn Hearnden, and Anna Forsythe are employees of Purple Squirrel Economics. 
      The authors report no other conflicts of interest in this work
EDAT- 2019/12/10 06:00
MHDA- 2019/12/10 06:01
PMCR- 2019/11/12
CRDT- 2019/12/10 06:00
PHST- 2019/08/07 00:00 [received]
PHST- 2019/10/09 00:00 [accepted]
PHST- 2019/12/10 06:00 [entrez]
PHST- 2019/12/10 06:00 [pubmed]
PHST- 2019/12/10 06:01 [medline]
PHST- 2019/11/12 00:00 [pmc-release]
AID - 226323 [pii]
AID - 10.2147/CEOR.S226323 [doi]
PST - epublish
SO  - Clinicoecon Outcomes Res. 2019 Nov 12;11:673-681. doi: 10.2147/CEOR.S226323. 
      eCollection 2019.